Department of Pharmacy, Fujian Medical University Union Hospital, Fuzhou, China.
The School of Pharmacy, Fujian Medical University, Fuzhou, China.
Support Care Cancer. 2022 Jul;30(7):6113-6121. doi: 10.1007/s00520-022-07041-2. Epub 2022 Apr 14.
Axicabtagene ciloleucel (Axi-Cel, 2 × 10 CAR-T cells/kg, single intravenous injection) is a chimeric antigen receptor cell immunotherapy that exhibits favorable clinical efficacy and safety in patients with relapsed or refractory diffuse large B-cell lymphoma (R/R DLBCL). However, this treatment is expensive in China. This study aimed to evaluate the cost-effectiveness of Axi-Cel versus salvage chemotherapy for the treatment of R/R DLBCL from the perspective of the Chinese healthcare system.
A decision analysis model containing a short-term decision tree and long-term semi-Markov partitioned survival model was developed. The time horizon was 40 years and the period from 10 to 40 years was included in sensitivity analysis. The model was developed based on data from the ZUMA-1 and SCHOLAR-1 trials. Life years, quality-adjusted life years (QALYs), overall costs, and the incremental cost-effectiveness ratio (ICER) were estimated at a willingness to pay (WTP) threshold of US $31,320 per QALY, which is three times the gross domestic product per capita.
The base case analysis revealed that treatment with Axi-Cel is associated with an increased overall cost of US $175,380 and improved effectiveness of 3.43 LYs and 2.61 QALYs compared to salvage chemotherapy, leading to an ICER of US $51,190 per LY and US $67,250 per QALY. The developed model is sensitive to the discount rate, utility of progression-free survival (PFS), and cost of Axi-Cel. The ICER of Axi-Cel was greater than the WTP threshold in the sensitivity and scenario analyses. To achieve cost-effectiveness, the price of Axi-Cel must be reduced by 59.19% to US $71,000.
At its current price, Axi-Cel is not likely to be a cost-effective option compared to salvage chemotherapy for adult patients with R/R DLBCL.
Axicabtagene ciloleucel(Axi-Cel,2×10 CAR-T 细胞/公斤,单次静脉注射)是一种嵌合抗原受体细胞免疫疗法,在复发或难治性弥漫性大 B 细胞淋巴瘤(R/R DLBCL)患者中显示出良好的临床疗效和安全性。然而,这种治疗在中国非常昂贵。本研究旨在从中国医疗保健系统的角度评估 Axi-Cel 与挽救化疗治疗 R/R DLBCL 的成本效益。
开发了一个包含短期决策树和长期半马尔可夫分区生存模型的决策分析模型。时间范围为 40 年,10 年至 40 年的时间段包含在敏感性分析中。该模型是基于 ZUMA-1 和 SCHOLAR-1 试验的数据开发的。在支付意愿(WTP)阈值为 31320 美元/QALY 的情况下,估计了生命年、质量调整生命年(QALY)、总成本和增量成本效益比(ICER)。
基础案例分析表明,与挽救化疗相比,使用 Axi-Cel 治疗可导致总费用增加 175380 美元,总生存获益增加 3.43 年,QALY 增加 2.61 年,导致每生命年 51190 美元,每 QALY 67250 美元的 ICER。所开发的模型对贴现率、无进展生存期(PFS)效用和 Axi-Cel 成本敏感。在敏感性和情景分析中,Axi-Cel 的 ICER 均高于 WTP 阈值。要实现成本效益,Axi-Cel 的价格必须降低 59.19%至 71000 美元。
与挽救化疗相比,目前 Axi-Cel 的价格对于 R/R DLBCL 成年患者来说不太可能是一种具有成本效益的选择。